Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised Phase 2 trial.
Burt RK, Shah SJ, Dill K et al.
Systemic sclerosis (SSc) is a chronic autoimmune disease in which there is little hope of improvement in the overall disease process. Hematopoietic stem cell transplantation (HSCT) is a potential treatment that relies on early intervention during the immune activation period. Previous studies of HSCT for SSc have shown significant improvements in the modified Rodnan skin score (mRSS), and two small studies have suggested an improvement in lung function (Ann Rheum Dis 2006;65:508–14, J Rheumatol 2009;36:1460–3). In this Phase II trial the safety and efficacy of autologous non-myeloablative HSCT were compared with the results in a control group that received one dose of intravenous cyclophosphamide every month for 6 months.